Based on positive phase III study data, Applied Therapeutics Inc. plans to take its CNS-penetrant aldose reductase inhibitor to the U.S. FDA to talk about an NDA for treating the rare disease sorbitol dehydrogenase (SORD) deficiency. Interim data from 12 months of treatment showed govorestat (AT-007) hit the study’s primary endpoints along with several key secondary endpoints. The double-blind, placebo-controlled registrational study of patients ages 16 to 55 is ongoing, with another 12 months of data yet to come. SORD, a hereditary axonal neuropathy created by sorbitol dehydrogenase gene mutations, affects about 3,300 people in the U.S. and about 4,000 in Europe, according to Applied Therapeutics.
Shares of Applied Therapeutics Inc. (NASDAQ:APLT) dropped 36% Jan. 5 on news that its oral aldose reductase inhibitor, AT-001 (caficrestat), failed to reach statistical significance in a phase III study in patients with diabetic cardiomyopathy. The New York-based firm, however, pointed to positive trends in the Arise-HF study that could offer a path forward, albeit via a partner.
After discussions with the FDA, Applied Therapeutics Inc. said it will again delay an NDA filing for its galactosemia treatment, AT-007, until it has more time to discuss the filing with the agency.
Applied Therapeutics Inc. (ATI) CEO Shoshana Shendelman said positive top-line findings with aldose reductase inhibitor AT-007 in galactosemia were “the data we needed, and we’re going to move as quickly as we can to get in and speak to the FDA about this, not only to firm up our NDA filing but also to get into the pediatric study.”